A Phase III, Multi-National, Double-Blind, Randomized, Placebo-Controlled, Stratified, Parallel Group Study to Evaluate the Safety, Tolerability and Efficacy of Tirasemtiv in Patients With Amyotrophic Lateral Sclerosis (ALS) (IRB no. 40131)
The purpose of this study is to evaluate the use of a drug called tirasemtiv (CK-2017357) as a potential new therapy for the improvement of breathing, muscle weakness and muscle fatigue in patients with amyotrophic lateral sclerosis (ALS). The study will examine the safety and effectiveness of tirasemtiv and how well it is tolerated in patients with ALS. Researchers aim to determine the effects of the drug on lung function, muscle performance and overall physical function, as well as any side effects.
Tirasemtiv is an investigational drug that has not been approved by the U.S. Food and Drug Administration.
EligibilityKey Inclusion Criteria:
- At least 18 years old
- A diagnosis of familial or sporadic ALS 24 months or less before screening
- Upright slow vital capacity (a respiratory measure) at least 70 percent of predicted for age, height and sex
- Able to swallow tablets without crushing
- Use of non-invasive positive pressure ventilation, mechanical ventilation via tracheostomy, or on any form of oxygen supplementation at the time of screening
- Patients with a diaphragm pacing system (DPS) at study entry or who anticipate DPS placement during the course of the study
- Body mass index of 20.0 kg/m2 or lower
Richard Lewis, MD